CA3093347A1 - Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism - Google Patents

Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism Download PDF

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Publication number
CA3093347A1
CA3093347A1 CA3093347A CA3093347A CA3093347A1 CA 3093347 A1 CA3093347 A1 CA 3093347A1 CA 3093347 A CA3093347 A CA 3093347A CA 3093347 A CA3093347 A CA 3093347A CA 3093347 A1 CA3093347 A1 CA 3093347A1
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CA
Canada
Prior art keywords
aav
sequence
capsid
aav9
hybrid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3093347A
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English (en)
French (fr)
Inventor
Isabelle Richard
Evelyne GICQUEL
Federico Mingozzi
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Institut National de la Sante et de la Recherche Medicale INSERM
Genethon
Universite D'Evry Val D'Essonne
Association Institut de Myologie
Sorbonne Universite
Original Assignee
Institut National de la Sante et de la Recherche Medicale INSERM
Genethon
Universite D'Evry Val D'Essonne
Association Institut de Myologie
Sorbonne Universite
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Application filed by Institut National de la Sante et de la Recherche Medicale INSERM, Genethon, Universite D'Evry Val D'Essonne, Association Institut de Myologie, Sorbonne Universite filed Critical Institut National de la Sante et de la Recherche Medicale INSERM
Publication of CA3093347A1 publication Critical patent/CA3093347A1/en
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/075Adenoviridae
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
CA3093347A 2018-04-05 2019-04-04 Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism Pending CA3093347A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18305399.0 2018-04-05
EP18305399 2018-04-05
PCT/EP2019/058560 WO2019193119A1 (en) 2018-04-05 2019-04-04 HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM

Publications (1)

Publication Number Publication Date
CA3093347A1 true CA3093347A1 (en) 2019-10-10

Family

ID=62063468

Family Applications (2)

Application Number Title Priority Date Filing Date
CA3093347A Pending CA3093347A1 (en) 2018-04-05 2019-04-04 Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
CA3134183A Withdrawn CA3134183A1 (en) 2018-04-05 2019-10-04 Peptide-modified hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism and increased muscle transduction

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3134183A Withdrawn CA3134183A1 (en) 2018-04-05 2019-10-04 Peptide-modified hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism and increased muscle transduction

Country Status (13)

Country Link
US (2) US12037362B2 (he)
EP (2) EP3775173A1 (he)
JP (2) JP7374119B2 (he)
KR (2) KR20210015770A (he)
CN (1) CN113795574A (he)
AU (2) AU2019249890A1 (he)
BR (2) BR112020020223A2 (he)
CA (2) CA3093347A1 (he)
IL (2) IL277740A (he)
MA (1) MA51353B1 (he)
TN (1) TN2020000187A1 (he)
WO (2) WO2019193119A1 (he)
ZA (1) ZA202005422B (he)

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CN112040988A (zh) * 2018-04-27 2020-12-04 海德堡大学 修饰的aav衣壳多肽用于治疗肌肉疾病
WO2021053124A1 (en) 2019-09-19 2021-03-25 Genethon Gene therapy expression system alleviating cardiac toxicity of fkrp
EP4073087A4 (en) * 2019-11-08 2023-11-29 The Board Of Regents Of The University Of Texas System RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR FOR GENE DELIVERY
TW202129002A (zh) * 2019-12-09 2021-08-01 英商Ucl商業有限責任公司 用於myh7關聯之心肌病之基因療法組合物及治療
US20230032826A1 (en) 2019-12-18 2023-02-02 Genethon Production of recombinant viral vectors from plant hairy roots
WO2021229255A1 (en) 2020-05-14 2021-11-18 Genethon Tools and method for preventing a a¥ neutralization by antibodies
EP3913060A1 (en) 2020-05-22 2021-11-24 Genethon Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy
EP4162049A1 (en) 2020-06-09 2023-04-12 Genethon Cilp-1 inhibitors for use in the treatment of dilated cardiomyopathies
EP4161559A1 (en) 2020-06-09 2023-04-12 Genethon Nrf2 activator for use in treating dilated cardiomyopathies
CA3185497A1 (en) 2020-06-09 2021-12-16 Genethon Treatment of genetic dilated cardiomyopathies
US20230256117A1 (en) 2020-06-19 2023-08-17 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
US20230242905A1 (en) 2020-07-03 2023-08-03 Genethon Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping
CA3184923A1 (en) 2020-07-10 2022-01-13 William LOSTAL A novel muscle-specific promoter
CN116096431A (zh) 2020-09-10 2023-05-09 吉尼松公司 肽修饰的aav衣壳
US20230272433A1 (en) 2020-09-29 2023-08-31 Genethon Enhancing Utrophin Expression in Cell by Inducing Mutations Within Utrophin Regulatory Elements and Therapeutic Use Thereof
WO2022204476A1 (en) 2021-03-26 2022-09-29 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
WO2022226301A1 (en) * 2021-04-23 2022-10-27 The University Of North Carolina At Chapel Hill Chimeric heart tropic aav capsids
JP2024529540A (ja) 2021-08-04 2024-08-06 ジェネトン 筋肉及びcnsにおける遺伝子発現のためのハイブリッドプロモーター
AU2023211652A1 (en) 2022-01-25 2024-08-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023237731A1 (en) 2022-06-09 2023-12-14 Genethon N-terminal truncated gde for the treatment of glycogen storage disease iii
WO2023237748A1 (en) 2022-06-10 2023-12-14 Genethon Peptide-modified aav capsid with enhanced muscle transduction efficiency
WO2024074727A1 (en) 2022-10-07 2024-04-11 Genethon Immunotherapy of skeletal myopathies using anti-fap car-t cells
WO2024079249A1 (en) 2022-10-12 2024-04-18 Genethon Hybrid aav vector enhancing transgene expression in the liver
CN116041443B (zh) * 2022-12-30 2023-09-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN116970041B (zh) * 2023-07-21 2024-02-09 广州派真生物技术有限公司 腺相关病毒突变体及其应用

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DK2292780T3 (en) 2003-09-30 2017-12-04 Univ Pennsylvania Clades and sequences of adeno-associated virus (AAV), vectors containing them, and uses thereof
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
ES2724800T3 (es) 2011-02-17 2019-09-16 Univ Pennsylvania Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
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HUE047996T2 (hu) 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
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US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
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Publication number Publication date
EP3775173A1 (en) 2021-02-17
TN2020000187A1 (en) 2022-04-04
JP2022526405A (ja) 2022-05-24
KR20210153047A (ko) 2021-12-16
CN113795574A (zh) 2021-12-14
BR112020020223A2 (pt) 2021-01-19
EP3947653A1 (en) 2022-02-09
JP2021520203A (ja) 2021-08-19
US20220228167A1 (en) 2022-07-21
IL277740A (he) 2020-11-30
IL286926A (he) 2021-10-31
CA3134183A1 (en) 2020-10-08
MA51353B1 (fr) 2022-09-30
BR112021019821A2 (pt) 2021-12-07
WO2019193119A1 (en) 2019-10-10
CN112004925A (zh) 2020-11-27
US20210107948A1 (en) 2021-04-15
AU2019249890A1 (en) 2020-09-24
ZA202005422B (en) 2023-03-29
RU2020136195A (ru) 2022-05-06
WO2020200499A1 (en) 2020-10-08
US12037362B2 (en) 2024-07-16
JP7374119B2 (ja) 2023-11-06
AU2019439673A1 (en) 2021-10-07
MA51353A1 (fr) 2021-09-30
KR20210015770A (ko) 2021-02-10

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EEER Examination request

Effective date: 20220831

EEER Examination request

Effective date: 20220831

EEER Examination request

Effective date: 20220831

EEER Examination request

Effective date: 20220831

EEER Examination request

Effective date: 20220831